NICE ‘no’ for BioMarin’s Vimizin
The draft guidance has been published following an evaluation of new evidence on the use of the treatment
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Tag: rare disease
HRA Pharma Rare Diseases funds online hub for Cushing’s Syndrome
Cushing’s Syndrome is rare and severe condition caused by prolonged high levels of cortisol in the blood
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Priority review for Merck & Co’s belzutifan in rare kidney cancer subset
Belzutifan is a potential treatment for von Hippel-Lindau disease-associated renal cell carcinoma
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Pharming’s APDS drug scores EU orphan status
European commission grants leniolisib orphan drug designation
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Healx, Boehringer link for rare neurological disease therapy
The project will utilise Healx’ AI-based drug discovery platform
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Shire, Microsoft and EURODIS form rare diseases alliance
Shire has teamed up with Microsoft and EURORDIS-Rare Diseases Europe in a strategic alliance aiming to tackle the diagnostic challenge for patients living with a rare disease.
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NICE deems Batten disease therapy too costly for NHS use
The National Institute for Health and Care Excellence has issued draft guidelines concluding that Biomarin’s Brineura is not a cost-effective treatment option for patients with the ultra rare disorder Batten disease.
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Shire mulls over separation of neuroscience unit
Shire is considering cleaving its neuroscience division into a separate, publicly-listed entity in a move that would afford the firm a sharper focus on rare diseases.
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- Liverpool and McMaster University announce £300,000 strategic research partnership
- Study finds link between malaria parasites developing antimalarial drug resistance
- Researchers develop ‘digital twin’ heart models to better monitor PAH NHS patients
- Closed Loop and Pharmanovia investigate precision medicine combination in hypertension
- US study reveals interferon combinations cause lupus symptoms and affect treatment
